Neuromuscular

The Bushell Chair of Neurology at the University of Sydney shared his insight into the latest data on CNM-Au8 as a potential treatment for amyotrophic lateral sclerosis and the frameshift in treatment that it represents. [WATCH TIME: 3 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending April 1, 2022.

The president and chief executive officer of The ALS Association provided insight on the most pressing needs for patients with ALS and the organization’s focus going forward. [WATCH TIME: 5 minutes]

FDA panelists expressed concern over the drug's efficacy and data analysis, indicating the need for a confirmatory clinical trial.

The investigational antisense oligonucleotide, previously known as IONIS-C9Rx, failed to meet any of the secondary efficacy end points in its phase 1 trial but was well-tolerated. The companies plan to present the data at a future medical meeting.

The president and chief executive officer of The ALS Association provided insight on the availability of genetic testing for ALS and the organization’s efforts to increase access for the future. [WATCH TIME: 2 minutes]

Abnormal scores on cognitive testing persisted in 50% of patients without a pre-COVID history of cognitive abnormalities, irrespective of the presence or absence of a neurological complication during hospitalization.

The president and chief executive officer of The ALS Association highlighted a few of the major developments in the ALS field over the past decade, led by advances in technology. [WATCH TIME: 3 minutes]

Here's what is coming soon to NeurologyLive®.

Test your neurology knowledge with NeurologyLive®'s weekly quiz series, featuring questions on a variety of clinical and historical neurology topics. This week's topic is headache and migraine.

Neurology News Network for the week ending March 26, 2022. [WATCH TIME: 4 minutes]

Take 5 minutes to catch up on NeurologyLive®'s highlights from the week ending March 25, 2022.

The president and chief executive officer of The ALS Association discussed how prevention is being talked about in the ALS community and why it’s no longer a far-off reality. [WATCH TIME: 4 minutes]

Catch up on any of the neurology news headlines you may have missed over the course of the last month, compiled all into one place by the NeurologyLive® team.

During Cerebral Palsy Awareness Month, leaders in the field expressed their love for the CP community, the progress made in managing the disorders, and the steps needed to advance care into a realm never before seen.

The senior investigator in the Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS shared his perspective on the data coming out of neuromuscular research and the promise of genetic medicine. [WATCH TIME: 2 minutes]

Topline data from the phase 3 ADAPT-SC study (NCT04735432) suggest that the subcutaneous formulation of the Argenx product was statistically noninferior to the recently approved IV formulation, efgartigimod afla-fcab (Vyvgart).

The president and chief executive officer of The ALS Association provided commentary on the most pertinent issues with managing patients with ALS and the ways to improve current treatment standards. [WATCH TIME: 4 minutes]

The president and chief executive officer of The ALS Association provided insight on how the organization is advancing the therapeutic pipeline of ALS agents for the foreseeable future. [WATCH TIME: 3 minutes]

The director of neuromuscular clinical trials at Columbia University Irving Medical Center shared her perspective on the discussions that took place at MDA 2022 and the importance of collaboration among clinicians and researchers.

The president and CEO of the Muscular Dystrophy Association spoke about the need for increased participation in neuromuscular disorder clinical trials and the rapid therapeutic progress the field is seeing. [WATCH TIME: 4 minutes]

The president and chief executive officer of The ALS Association expanded on the organization’s greatest goal—making ALS livable by 2030—and how it plans to work towards that over the next decade. [WATCH TIME: 3 minutes]

Eleven of the 12 individuals demonstrated at least a 50% reduction in creatine kinase, with 75% of participants reaching twice the normal range, suggesting a reduction in muscle breakdown.

More than 90% of the 65-patient cohort had evidence of normal swallowing and had no aspiration or pneumonia aspiration events reported in the phase 1 START and phase 3 STRIVE-EU and STRIVE-US trials.

In terms of cardiomyopathy, disease-modifying bioactivity of cardiosphere-derived cells was shown by improvements in left ventricular ejection fraction, a commonly used measure of global heart function.